ASGCT 2021 | Synthetic AAV vectors for gene therapy of hemophilia in children

Arun Srivastava, PhD, University of Florida, Gainesville, FL, explains that a key challenge associated with the use of adeno-associated virus (AAV) vectors for gene therapy for hemophilia in children is the fact that up until age 10-12, the liver is still growing and dividing, meaning traditional AAV gene therapies are diluted out with every cell division, given their episomal nature. Synthetic AAV vectors containing a no-end (NE) AAV DNA encapsulated in liver-specific synthetic liposomes are currently being investigated. It is hoped that this strategy may allow repeated dosing and specific targeting of the liver, without the induction of a host immune response. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.